Gene and base editors deliver on fetal hemoglobin expression: ASH24

In the endeavor to cure sickle cell disease with a one-time genetic treatment, partners Vertex and CRISPR Therapeutics are the field’s leaders, but new data presented at ASH provide early signals that up-and-coming clinical candidates could have the efficacy edge.

It’s been one year since the approvals of the first two genetic medicines for sickle cell disease: Casgevy exagamglogene autotemcel from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) and CRISPR Therapeutics AG (NASDAQ:CRSP), and Lyfgenia lovotibeglogene autotemcel from bluebird bio Inc. (NASDAQ:BLUE). ...